Y-mAbs Reveals New Phase 2 Data for Naxitamab at 2024 ASCO Meeting

Y-mAbs Therapeutics, Inc. (Nasdaq: YMAB), a commercial-stage biopharmaceutical company, has released new interim data from its Phase 2 Trial 201, evaluating the efficacy of naxitamab combined with granulocyte-macrophage colony-stimulating factor (GM-CSF) in patients with relapsed or refractory high-risk neuroblastoma confined to bone and/or bone marrow. These results were presented on June 1, 2024, at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago.

The poster, titled “Patterns of improvement following initial response in patients treated with naxitamab for relapsed/refractory high-risk neuroblastoma” (poster #10033), indicates that extended naxitamab therapy can further mitigate the disease following an initial positive response. The interim analysis of Trial 201 revealed a 50% overall response rate (ORR) in 26 out of 52 patients. About 25% of patients experienced their first complete or partial response after undergoing three or more cycles of naxitamab therapy. Most of these patients initially had stable disease in the bone or bone marrow before achieving a complete or partial response. These findings advocate for the continuation of naxitamab therapy in patients who do not initially achieve a complete or partial response.

Another poster, “Naxitamab-related adverse events within and across treatment cycles in patients with relapsed/refractory high-risk neuroblastoma” (poster #10032), discusses the frequency and patterns of adverse events (AEs) from the interim analysis. Approximately 81% of naxitamab-related AEs were Grade 1 or 2. Grade 3 or higher AEs identified in at least 10% of patients included hypotension (60% of patients), pain (58%), urticaria (19%), bronchospasm (18%), and abdominal pain (16%). None of these AEs led to treatment discontinuation. Notably, the incidence of Grade 3 naxitamab-related pain decreased from 53% in the first cycle to 37% in the second cycle, stabilizing across subsequent cycles. Hypotension occurrences also significantly reduced over the treatment cycles.

Dr. Vignesh Rajah, Chief Medical Officer, expressed optimism, noting that some patients who did not respond initially went on to achieve a complete or partial response with continued treatment. The data also provide insights into the temporal patterns of treatment-related adverse events.

In addition to Y-mAbs' presentations, Dr. Jaume Mora and his team from the Sant Joan de Déu Barcelona Children’s Hospital presented a poster titled “Naxitamab chemo-immunotherapy regimens other than with irinotecan/temozolomide for patients with relapsed/refractory high-risk neuroblastoma” (poster #10037). This independent retrospective analysis involved 29 patients with chemo-immunotherapy refractory disease who received compassionate use of naxitamab plus sargramostim combined with alternative chemotherapeutics. Prior sensitivity to chemo-immunotherapy predicted better outcomes with these new combinations, and the safety profiles were deemed manageable.

DANYELZA® (naxitamab-gqgk), developed by researchers at Memorial Sloan Kettering Cancer Center (MSK) and exclusively licensed to Y-mAbs, is indicated for treating pediatric and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have previously shown a partial response, minor response, or stable disease. This indication was approved under accelerated approval based on overall response rate and response duration. However, continued approval may be contingent on confirmatory trials.

Y-mAbs focuses on developing and commercializing innovative radioimmunotherapy and antibody-based treatments for cancer. Their technology portfolio includes the Self-Assembly DisAssembly (SADA) Pretargeted Radioimmunotherapy Platform and bispecific antibodies generated using the Y-BiClone platform. Their product pipeline includes the FDA-approved anti-GD2 therapy DANYELZA® for relapsed or refractory high-risk neuroblastoma.

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